Autosomal-dominant polycystic kidney disease (ADPKD) is one of the most common genetic disorders, affecting one in every 1,000 people and responsible for up to ten percent of patients on dialysis worldwide. The disease is characterized by the development of cysts that lead to progressive kidney failure and necessitate dialysis or a kidney transplant in most patients aged around fifty. Moreover, the persistent cyst growth causes high blood pressure and painful complications. Although we have known about the disease for over a century and its genetic basis for almost 20 years, there was no effective treatment until now.
Kidneys stopped growing
"Our study highlights a potential treatment that reduces kidney growth and the associated symptoms and slows the decline in kidney function," explains Professor Olivier Devuyst from the Institute of Physiology at the University of Zurich - one of the chief researchers in the phase-three clinical trial just published in the New England Journal of Medicine.
Over 1,400 patients were given tolvaptan over a three-year period at 129 centers worldwide. The drug is a selective V2 vasopressin receptor antagonist that lessens the effect of the antidiuretic (urine-concentrating) vasopressin hormone and increases urination.